A Phase I/II Open-label Study of Eltrombopag for the Prevention of Chemotherapy Induced Thrombocytopenia (CIT) in Subjects With Advanced Soft Tissue and Bone Sarcomas Receiving Gemcitabine and Docetaxel Chemotherapy
The combination chemotherapy regimen of gemcitabine and docetaxel has become an increasingly
used treatment choice for subjects with advanced sarcomas. The regimen has shown activity
in first and second line for subjects with metastatic uterine leiomyosarcoma,
relapsed/refractory pediatric sarcomas, and improved progression-free survival and overall
survival in persons with metastatic sarcoma when compared to gemcitabine alone. The regimen
has additionally been recognized as a treatment option for subjects with advanced sarcoma.
Chemotherapy-induced toxicity in the blood such as low platelet level is a common and often
therapy-limiting side effect of treatment.
There are two phases in this study. The purpose of the Phase I study is to determine the
recommended dose of eltrombopag. The purpose of the Phase II study is to determine the
safety, tolerability, and efficacy (how well the drug works) of eltrombopag in subjects who
are receiving gemcitabine and docetaxel chemotherapy.
Eltrombopag (Promacta) is an FDA approved drug for the treatment of chronic idiopathic
thrombocytopenic purpura (ITP)- a condition of having an abnormally low platelet count.
Eltrombopag is now being further investigated for other thrombocytopenic (low
Participants will take the assigned dose of eltrombopag once a day starting five days before
each cycle of chemotherapy and for 5 days after chemotherapy. Eltrombopag is not taken on
the day of chemotherapy. Eltrombopag is taken orally.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention
Tolerated Eltrombopag Dose
Measured in milligrams (mg). The primary objective of the phase I study is to determine the recommended phase II dose. The maximum dose is the dose of which less than 2 of 6 patients experienced an unacceptable event/side effect
Richard F Riedel, MD
United States: Food and Drug Administration
|Duke Cancer Center||Durham, North Carolina 27710|